Gates Foundation backs a new sickle cell CRISPR approach; Siddhartha Mukherjee's Vor adds a new CAR-T
CRISPR/Cas9 player Intellia Therapeutics has a new backer — the Bill and Melinda Gates Foundation.
The charity is sponsoring research for in vivo sickle cell disease treatments using their gene-editing technology. Financial terms for the grant were not disclosed, but the funds will be used to advance Intellia’s preclinical programs for editing hematopoietic stem cells using the company’s proprietary non-viral delivery systems.
Intellia’s long-term goal is to potentially cure sickle cell disease. CRISPR sickle cell treatments are already in the clinic, but they involve removing cells from patients, editing them and then putting them back in — a costly approach that also involves subjecting patients to high doses of chemotherapy. An in vivo approach could be safer but is considered harder to achieve.
The grant is part of an initiative from the foundation that aims to support gene-based cures in developing countries within the next seven to 10 years. — Max Gelman
Siddhartha Mukherjee’s Vor adds an NCI CAR-T
Vor, the cell therapy startup from Columbia oncologist and Pulitzer Prize-winning author Siddhartha Mukherjee, has added CAR-T to pair with its line of cell therapies.
On Wednesday, the Cambridge biotech announced they licensed a CAR-T therapy from the National Cancer Institute. Devised by well-known hematologist Terry Fry — who is now head of T cell therapies at the secretive, well-financed Sana Biotechnology — the therapy is now in Phase I/II for acute myeloid leukemia.
It aims at CD33, a cell receptor that AML researchers have long tried to target. Vor, has been developing a stem cell therapy in which they edit CD33 out of healthy cells and then transplant them into a patients bone marrow. The idea is that you could then give the CD33 CAR-T without risking the T cells attacking healthy cells along with cancer ones.
— Jason Mast
Another Compass goes public
Compass Pathways made waves earlier this year when it raised $127.5 million going public on the promise of turning the key ingredient in magic mushrooms into a treatment for depression.
Now another nautical named company — Compass Therapeutics — is following them onto the Nasdaq. They set terms this week for a $50 million IPO.
A much more conventional company, Compass Therapeutics is developing immuno-oncology treatments. Their lead drug, an antibody targeting CD137, is now in Phase I for patients who have previously taken PD-1 or PD-L1 inhibitors. They’ve raised $176.5 million on private markets so far, according to Crunchbase. — Jason Mast